FIELD: biotechnology.
SUBSTANCE: recombinant adeno-associated virus (AAV) capsid protein is described that is a hybrid of the capsid proteins of AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74), comprising a sequence having at least 85% identity with the following SEQ ID NO: 3, and has a reduced tropism for liver tissue compared to the capsid proteins of the original serotypes AAV9 and AAVrh74. The following is presented: a polynucleotide encoding the capsid protein of a recombinant hybrid AAV in an expressed form. Also, the following is presented: an AAV-based vector particle for expression of the desired gene in muscle cells containing a nucleotide sequence corresponding to the capsid protein of the recombinant hybrid AAV.
EFFECT: invention expands the arsenal of agents for genetic therapy.
10 cl, 4 dwg, 3 ex
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|
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